HD Clinical Research:
Getting Involved
HDSA Center of Excellence at UC Davis
Vicki Wheelock, M.D.
Terry Tempkin, RN-C, ANP
HD Clinical Research
Clinical research studies, especially
clinical trials, are how new knowledge is
generated, and they help to find new
treatments. Being a research participant is
not for everyone, but without participants
there cannot be new treatments in the
future.
Source: The Huntington Study Group Web Site
HD Clinical Research
Questions
 What is clinical research?
 What is an observational trial?
 Who can participate in research studies?
 How much time does it require?
 What about confidentiality?
 How do people decide whether to volunteer ?
 Are we making any progress?
 Which HD clinical trials are enrolling right now?
HD Clinical Research
HD Clinical Research: Definition
 The goal of clinical research is to study information
collected about the people enrolled in order to learn
about the manifestations of a disease, or to test the
safety, benefit, side effects and risks of an
intervention designed to help people affected by a
disease.
 Types of clinical research:
 Observational studies
 Clinical trials
• Double blind, randomized control trials
• Phase I, II, III
HD Clinical Research
The Pathway to Finding New Drugs
Pre-clinical: Models
Phase I: Test in healthy humans
for safety
Phase II: Test in small population
with disease for safety and dosing
Phase III: Test in larger
population for efficacy
FDA Approval
HD Clinical Research
What Are Observational Trials?
 These are studies that follow research
participants over time without making a treatment
intervention.
 Observational studies allow researchers to study
the progression of HD over time in a number of
individuals, and help us to understand the
symptoms people experience, the signs noticed
by investigators, the rate of progression, and
sometimes markers of HD such as brain scan or
blood test changes.
HD Clinical Research
What Are Double Blind, Randomized,
Controlled Studies?
 Designed to help prevent
judgment or bias by the
investigators or research
subjects from affecting the
study results.
 Double blind: neither the
investigator nor the
participant can tell if the
participant is taking the
active drug or a placebo.
HD Clinical Research
What Are Double Blind, Randomized,
Controlled Studies?
Randomized: The study participant
is assigned randomly to either
active drug or placebo.
Controlled: The study drug is
compared to a known treatment
(active control) or to a placebo
(inactive compound that appears
identical to the active drug).
HD Clinical Research
Who Can Participate In Clinical Trials?
 The study protocol is a research plan or roadmap
designed to best answer the question posed by the
study.
 The protocol includes criteria for who is eligible. This
could include:




People with the HD gene expansion who don’t yet have symptoms
People with HD at early, mid- or late-stage
People at 50% risk for the HD CAG expansion
Family members who are not at risk for HD, or people who have
undergone predictive testing and don’t have the HD CAG
expansion
HD Clinical Research
Who Can Participate In Clinical Trials?
Persons with:
 Ability to give informed consent

Or research proxy (person you designate to make research
decisions for you)
 Who meet age criteria
 Who have characteristics of HD: early, mid-stage, etc
 Other medical conditions
 Safety labs
 Psychiatric conditions
 Must be stable
 Other medications

Some may not be allowed
HD Clinical Research
How Much Time Is Required?
Studies vary in duration, frequency of study visits
and in the number of activities carried out.
 Observational studies can last many years

Visits are usually just once a year
 Clinical trials could last a few weeks to several years
 Visits are frequent for Phase II trials (every 1-4 weeks)
 Visits every 1 – 6 months in longer Phase III studies
 Initial study visits usually last 2 - 3 hours
 Follow-up visits may take as little as 15 minutes, or
as long as several hours
 Sometimes there may be telephone contacts made
between visits
HD Clinical Research
What Kinds Of Activities Take Place
During Study Visits?
 Initial visit: informed consent
 Principles of protection of human subjects require
approval of Institutional Review Boards or Ethics
Committees.
 Informed consent includes a description of the aims of
the study, the procedures that will take place, the
potential benefits, side effects and risks to participants.
 Participants are invited to ask questions for clarification
 Often 18 pages long
 Takes about 45 minutes
HD Clinical Research
What Kinds Of Activities Take Place
During Study Visits? (Cont.)
 Measuring “vital signs”: blood pressure, pulse,
weight
 Drawing blood samples
 Meet with site investigator to ask how participant
is doing, any health changes, any side effects,
perform physical and neurological exam
HD Clinical Research
What Kinds Of Activities Take Place
During Study Visits? (Cont.)
 Meet with site coordinator to ask
about mood or behavior changes,
test thinking, turn in empty study
medication bottles and dispense
study drug
 MRI brain scan
 Cognitive tests (memory, thinking,
etc)
HD Clinical Research
What About Privacy?
 Confidentiality is a prime concern to the HSG
 Participant identities are known to study site personnel,
but NOT to anyone else involved in the study
 Participants are assigned an ID code at the sites; all
communications with the central site use ID codes, not
names
 Blood samples are assigned bar codes to further protect
privacy
 To date, there have been no inadvertent disclosure of
identity or gene status
H·S·G
Huntington · Study · Group
HD Clinical Research
What Happens After
A Clinical Trial Is Over?
 The Huntington Study Group is committed to
communicating the results of completed clinical trials
to participants
 Timing: as soon as the study data is “locked”
 Mechanism:
 1st: conference call with participants
 HSG website, press releases
 Learning whether participant was assigned to active
drug or control group may take weeks or months
longer.
H·S·G
Huntington · Study · Group
HD Clinical Research
Previous HD Observational Studies
Study
PHAROS
1999 -2010
PREDICT
2001-2013
COHORT
2006-2011
Number of
Volunteers
1001
~1000
>2200
Target Participants
People at 50% risk for HD CAG expansion
who chose NOT to undergo predictive testing,
followed every 9 months for 10 years
People who have undergone testing for the
HD CAG expansion, followed annually since
2001
People who either have manifest HD, are at
50% risk, their family members, spouses who
are not at risk , followed annually 2004-2011
H·S·G
Huntington · Study · Group
HD Clinical Research
Examples of Completed
Clinical Trials
Study
Number of
Volunteers
Medication
TETRA-HD
84
Tetrabenazine
Positive – FDA
approved 2008
TREND-HD
316
Ethyl-EPA
(Fish oil)
Not effective
Horizon
300
Dimebon
Not effective
DOMINO
100
Minocycline
Not effective
Results
H·S·G
Huntington · Study · Group
HD Clinical Research
Getting Involved In Research:
Five Good Reasons For Participating
*From Terry Tempkin*
1. You’ve always wanted to help
2. You want to contribute to the efforts for a cure
3. You have the time
4. You care about HD research and future
generations
5. Participating in research makes you feel good
about helping to advance treatment and find a
cure
HD Clinical Research
Reasons For NOT Participating
In Clinical Research
 I don’t have enough time
 Work, school, family activities
 Caregiving for family members
 I’m concerned about my privacy.
 It’s too stressful to think about HD.
 I don’t want to have physical exams or thinking
tests; I’m already worried enough.
 I hate having blood tests!
 It’s too far to drive to the nearest study site.
 I don’t want to be on a placebo.
HD Clinical Research
There are reasons for and against
volunteering for clinical research….
HD Clinical Research
Enroll-HD
Longitudinal Observational Trial
The Goal:
 To create opportunities for research participation
for HD families on a global scale
 Through this effort, ENROLL- HD is creating a
repository of unified clinical information and
bio-samples critical for HD scientists world-wide.
HD Clinical Research
210 clinical sites across 28
countries....
Sponsored by CHDI Foundation, Inc. 2011
HD Clinical Research
Why Is This Important?
 This repository of data creates a wealth of
information to better understand the disease and
provide an important tool for future HD studies.
 Often studies require large numbers of
individuals in order to be sufficiently powerful
 Enroll-HD investigators will be collaborating from
across the globe to provide those large numbers
of participants
HD Clinical Research
Our New Hope For HD:
Stem Cell Research
 Dr. Jan Nolta
 Dr. Gerhard Bauer
HD Clinical Research
From Dr. Jan Nolta
HD Clinical Research
HD patients and families advocate for
stem cell research at CIRM meetings
HD Clinical Research
July 26, 2012
CIRM grant DR2A-05415 approved!
HD Clinical Research
We need your help. Please consider
volunteering for HD research.
HD Clinical Research
For More Information….
Amanda Martin (916) 734-3541
amanda.martin@ucdmc.ucdavis.edu
Terry Tempkin (916) 734-6278
teresa.tempkin@ucdmc.ucdavis.edu